Orchard Therapeutics Announces Multiple Presentations at ESGCT 2023
24 Outubro 2023 - 8:00AM
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader,
today announced eight presentations from across its hematopoietic
stem cell (HSC) gene therapy platform will be featured at the
European Society of Gene and Cell Therapy (ESGCT) 30th Annual
Congress taking place October 24-27, 2023, in Brussels.
Featured presentations include a range of
interim clinical outcomes in addition to the neurological and
skeletal results previously reported from the company’s
proof-of-concept study of OTL-203 in the Hurler subtype of
mucopolysaccharidosis type I (MPS-IH), as well as well as a first
look at pre-clinical data demonstrating the use of vectorized HSCs
as a delivery vehicle for monoclonal antibodies. In addition,
several abstracts from the company’s pre-clinical research programs
demonstrate the therapeutic potential of HSC gene therapy to
address larger indications, including a genetic sub-type of
frontotemporal dementia (FTD) and Crohn’s disease, as well as
chronic autoimmune disorders.
The company will also host a sponsored symposium
on Thursday, October 26, 2023, from 1:30 to 2:50 p.m. CEST
featuring expert speakers discussing the potential of HSC gene
therapy as a platform to deliver therapeutic genes to target organs
such as the brain, bone and GI tract for the treatment of more
prevalent diseases where current therapies are limited or do not
exist.
Additionally, Nicoletta Loggia, Ph.D., chief
technical officer, will give an invited talk on Friday, October 27
at 8:30 a.m. CEST, titled “Meeting the moment: Overcoming
manufacturing bottlenecks and technical challenges to usher a new
era of genetic medicines,” exploring strategies and innovations
critical to enabling the application of HSC gene therapy to address
larger indications.
“Together with our clinical collaborators, we’re
proud of our significant presence at ESGCT which continues to
underscore the transformative impact and broad applicability of our
HSC gene therapy platform,” said Fulvio Mavilio, Ph.D., chief
scientific officer of Orchard Therapeutics. “Collectively, these
data demonstrate the potential of our approach to correct the
underlying genetic defects responsible for several
difficult-to-treat diseases, furthering our efforts toward
unlocking the full promise of our portfolio.”
Details of the oral presentations are follows
(all times in CEST):
- Title: Lentiviral ex-vivo HSC gene
therapy as a tool to deliver therapeutic antibodies beyond the
blood-brain barrierDate/Time: Tuesday, October 24 at 6:15 p.m.Lead
Author: Chiara Recchi
- Title: Interim skeletal outcome
after hematopoietic stem and progenitor cell-gene therapy for
Mucopolysaccharidosis type I HurlerDate/Time: Wednesday, October 25
at 3:30 p.m.Lead Author: Maria Ester Bernardo
- Title: Development of an ex vivo
hematopoietic stem cell Gene Therapy for Frontotemporal
Dementia (FTD) Date/Time: Friday, October 27 at 9:45 a.m.Lead
Author: Yuri Ciervo
Details of the poster presentations are as
follows (all times in CEST):
- Title: Hematopoietic Stem Cell Gene
Therapy as a Novel Gene Therapy Approach for Severe Crohn’s Disease
Associated with NOD2-Deficiency Date/Time: Wednesday, October 25 at
5:00 p.m.Lead Author: Florence Enjalbert
- Title: HSC-derived CAR-Treg gene
therapy: a novel approach for the treatment of chronic autoimmune
diseaseDate/Time: Wednesday, October 25 at 5:00 p.m.Lead Author:
Greg Crawford
- Title: Interim non-neurological,
non-skeletal outcomes after hematopoietic stem and progenitor
cell-gene therapy for Mucopolysaccharidosis type I HurlerDate/Time:
Wednesday, October 25 at 6:15 p.m.Lead Author: Francesca Tucci
- Title: Dissecting the mechanisms of
myeloid-to-neural enzymatic cross-correction in the context of
hematopoietic stem cell gene therapy for Metachromatic
Leukodystrophy Date/Time: Wednesday, October 25 at 5:00 p.m.Lead
Author: Vasco Meneghini
- Title: Downstream strategies for
concentration and purification of LV Vectors from suspension
cell-based systemsDate/Time: Wednesday, October 25 at 6:15 p.m.Lead
Author: Luca Crippa
About Orchard TherapeuticsAt
Orchard Therapeutics, our vision is to end the devastation caused
by genetic and other severe diseases. We aim to do this by
discovering, developing and commercializing new treatments that tap
into the curative potential of hematopoietic stem cell (HSC) gene
therapy. In this approach, a patient’s own blood stem cells are
genetically modified outside of the body and then reinserted, with
the goal of correcting the underlying cause of disease in a single
treatment.
In 2018, the company acquired GSK’s rare disease
gene therapy portfolio, which originated from a pioneering
collaboration between GSK and the San Raffaele Telethon Institute
for Gene Therapy in Milan, Italy. Today, Orchard is advancing a
pipeline spanning pre-clinical, clinical and commercial stage HSC
gene therapies designed to address serious diseases where the
burden is immense for patients, families and society and current
treatment options are limited or do not exist.
Orchard has its global headquarters
in London and U.S. headquarters in Boston. For
more information, please visit www.orchard-tx.com, and follow
us on X (Twitter) and LinkedIn.
Availability of Other Information About
OrchardInvestors and others should note that Orchard
communicates with its investors and the public using the company
website (www.orchard-tx.com), the investor relations website
(ir.orchard-tx.com), and on social media (X;
Twitter and LinkedIn), including but not limited to
investor presentations and investor fact sheets, U.S.
Securities and Exchange Commission filings, press releases,
public conference calls and webcasts. The information that Orchard
posts on these channels and websites could be deemed to be material
information. As a result, Orchard encourages investors, the media,
and others interested in Orchard to review the information that is
posted on these channels, including the investor relations website,
on a regular basis. This list of channels may be updated from time
to time on Orchard’s investor relations website and may include
additional social media channels. The contents of Orchard’s website
or these channels, or any other website that may be accessed from
its website or these channels, shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933.
Forward-looking StatementsThis
press release contains forward-looking statements, which are made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. All statements that are not
statements of historical facts are, or may be deemed to be,
forward-looking statements. Forward-looking statements include
express or implied statements relating to, among other things, the
therapeutic potential of Orchard’s products and product candidates.
These statements are neither promises nor guarantees and are
subject to a variety of risks and uncertainties, many of which are
beyond Orchard’s control, which could cause actual results to
differ materially from those contemplated in these forward-looking
statements. In particular, these risks and uncertainties include,
without limitation, the risk that products will not be successfully
commercialized, and the risk that long-term adverse safety findings
may be discovered. Given these uncertainties, the reader is advised
not to place any undue reliance on such forward-looking
statements.
Other risks and uncertainties faced by Orchard
include those identified under the heading "Risk Factors" in
Orchard’s most recent annual or quarterly report filed with the
U.S. Securities and Exchange Commission (SEC), as well as
subsequent filings and reports filed with the SEC. The
forward-looking statements contained in this press release reflect
Orchard’s views as of the date hereof, and Orchard does not assume
and specifically disclaims any obligation to publicly update or
revise any forward-looking statements, whether as a result of new
information, future events or otherwise, except as may be required
by law.
Contact
Benjamin Navon
+1 857-248-9454
Benjamin.Navon@orchard-tx.com
Orchard Therapeutics (NASDAQ:ORTX)
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